We shared challenging news with the Duchenne community in September 2023. The European Medicine Agency’s (EMA) human medicines committee (CHMP) released a recommendation (read more here) against renewing the approval for the marketing of Translarna in the European Union, which had allowed Translarna to be made available to EU patients.
Despite efforts by the manufacturer, PTC, to appeal and request a formal re-examination, the EMA confirmed its decision on Friday, January 26, 2024. It maintains the recommendation for non-renewal of the marketing authorisation for Translarna. This EMA reviewed findings that show evidence of benefit from the treatment, but concluded that those findings were not statistically significant. Importantly, there are no safety concerns associated with the treatment.
Unfortunately, for patients in the EU, this means that Translarna will soon no longer be available.
Crucially, this decision does not impact availability in England, Wales, and Scotland, as marketing authorization in Great Britain falls under the responsibility of the Medicines and Healthcare products Regulatory Agency (MHRA) rather than the EMA. We seek clarification on availability in Northern Ireland and will provide any updates as soon as they are issued.
Translarna continues to be available in Scotland through the ‘ultra-orphan pathway,’ and the Scottish Medicines Consortium (SMC) is scheduled to appraise it in 2025.
In January 2023, the National Institute of Health and Care Excellence (NICE) published final guidance recommending Translarna as a treatment option for Duchenne muscular dystrophy resulting from a ‘nonsense mutation’ and acknowledged the positive impact of the treatment on the lives of both patients and caregivers.
Action Duchenne remains fully supportive of the treatment’s continued availability and are disappointed by the outcome of the EMA process. We stand firmly committed to supporting and advocating for those affected by Duchenne muscular dystrophy. As we receive further information, we will keep you updated on developments.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
SAVE THE DATE
Our 2024 Annual International Conference will be held on Friday 8th and Saturday 9th November 2024. The conference brings together families, clinicians, researchers and experts for 2 days of sharing knowledge and experience. It is an amazing opportunity to come together as part of the Duchenne community
